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Evolving therapies for lower-risk myelodysplastic syndromes.
Annals of Hematology ( IF 3.5 ) Pub Date : 2020-02-20 , DOI: 10.1007/s00277-020-03963-1
Jan Philipp Bewersdorf 1 , Amer M Zeidan 1
Affiliation  

The development in the therapeutic landscape of myelodysplastic syndromes (MDS) has substantially lagged behind other hematologic malignancies with no new drug approvals for MDS for 13 years since the approval of decitabine in the United States in 2006. While therapeutic concepts for MDS patients continue to be primarily defined by clinical-pathologic risk stratification tools such as the International Prognostic Scoring System (IPSS) and its revised version IPSS-R, our understanding of the genetic landscape and the molecular pathogenesis of MDS has greatly evolved over the last decade. It is expected that the therapeutic approach to MDS patients will become increasingly individualized based on prognostic and predictive genetic features and other biomarkers. Herein, we review the current treatment of lower-risk MDS patients and discuss promising agents in advanced clinical testing for the treatment of symptomatic anemia in lower-risk MDS patients such as luspatercept and imetelstat. Lastly, we review the clinical development of new agents and the implications of the wider availability of mutational analysis for the management of individual MDS patients.

中文翻译:

不断发展的低风险骨髓增生异常综合症疗法。

自2006年美国地西他滨获批以来,骨髓增生异常综合征(MDS)治疗领域的发展已远远落后于其他血液系统恶性肿瘤,至今已有13年没有MDS的新药批准。尽管MDS患者的治疗概念仍在继续主要由临床病理风险分层工具(例如国际预后评分系统(IPSS)及其修订版IPSS-R)定义,在过去的十年中,我们对MDS的遗传格局和分子发病机制的理解有了很大的发展。预期基于预后和预测性遗传特征以及其他生物标记物,针对MDS患者的治疗方法将变得越来越个性化。在这里 我们回顾了低危MDS患者的当前治疗方法,并讨论了在高级临床测试中用于治疗低危MDS患者(如luspatercept和imetelstat)症状性贫血的有希望的药物。最后,我们回顾了新药的临床开发以及突变分析对个体MDS患者管理的广泛应用的意义。
更新日期:2020-02-20
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